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CBS News
16 hours ago
- Health
- CBS News
Bay Area family shares frustrations after FDA rejects drug for ultra-rare Barth Syndrome
With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments in the rare disease community. Approximately 150 people live with Barth Syndrome nationwide, an ultra-rare genetic disorder that affects only males and impacts muscle and heart muscle health. "He has a severe heart failure, and was admitted to the hospital where he was on intravenous medications to help his heart pump," Megan Branagh, whose son Henry has Barth Syndrome, told CBS News Bay Area. Megan's son was diagnosed with the disease when he was three months old. "There's no treatment or cure for it at all, just simply symptom management," she said. There are currently no FDA-approved therapies for the disease, but Stealth BioTherapeutics has been working for years to get a target drug approved. "Extremely long and delayed," Megan said. "We were supposed to get an answer in January, and FDA asked for a three-month extension based on some data they had requested." The drug, elamipretide, targets cardiolipins, a molecule in the mitochondria that is a key source for the gene defect. It's the only drug for Barth Syndrome clinical trials. After several delays, however, the FDA announced it will not be approving the drug application. Megan believes federal budget cuts could be one of the reasons. "There was only one person left remaining at the FDA who had a hand on our case for this final part of it," she said. "We've heard from the current administration the desire to make medication available for the rare disease community, and the support of the rare disease community. And this case defies everything that has been publicly stated over the last few months." The FDA instead recommended that more data be included in the company's resubmission of the drug application. While the company said they are optimistic for a new path forward with the agency, families like the Branaghs are anxiously waiting for the approval. "Sickening to know that there's something out there that could make a difference that might never be available for Henry or anyone else," she said. Her son is 13 years old and has endured many challenges, taking at least eight pills a day and making frequent trips to the doctor's office. "I do it and I just get it over with," Henry Branagh told CBS News Bay Area. "And I wait six more months, and they usually put a heart monitor for two days and I take it off." He shared that he enjoys playing sports, including baseball, soccer, skiing, and swimming. "I love playing with my friends," Henry said. He added that he does take more frequent breaks to maintain his heart health, and hopes there will be more treatment options for him soon. "It'd be really nice for me to have it too, because my wish is to like live one day be normal without having Barth Syndrome," Henry said. "Mainly when I'm in sports, and I'm running around, and I get tired and other kids are running way faster and have way better stamina." Dr. Hitenda Patel, the Branagh's family cardiologist, told CBS News Bay Area that he doesn't see the application rejection as a denial. "I think the FDA wants a little more data on its efficacy," Patel, who is also the pediatric cardiology director at UCSF Benioff Children's Hospital in Oakland, said. "There are a lot more designer drugs being developed. And designer drugs specifically, are often for rare diseases. And if you only have, let's say, at the most 500 patients in the world, it's very hard to get approval based on the current mandates for the FDA," he added. Patel said FDA approvals in the pediatric rare disease community are an even bigger hill to climb. "It seems like it's a delaying tactic, but I think you have to respect the FDA and its process because it's what has allowed to develop many things," he said. "And ultimately, they are in charge of the safety of the American people." He said that with work from those like the Branagh's do make an impact. "With advocacy such as the Barth Syndrome and what the Branaghs are doing, FDA has realized that there is a huge difference between approval process that is needed for the pediatric and rare disease market as opposed to something that is more common," Patel said. "Because the FDA has its mandates and you have to go through the trial process, it takes a long, long, long time." Patel said he remains optimistic for the community that often gets overlooked. And so, the fight continues for the Branaghs. "Super proud. He inspires us to live our best every moment and take nothing for granted," Henry's mother said. "I think the worst thing to watch as a parent to know that your kid has something that you can't do anything about." She adds that she will continue to fight for his health, no matter how long it takes. "My parents help me out a lot, they just always push me and encourage me to keep on going," Henry said. CBS News Bay Area reached out to Stealth BioTherapeutics, and a spokesperson said that while there is no public timeline yet for next steps, they are working closely with the FDA urgently to reach approval. Meanwhile, the Branaghs are preparing for their next fundraising campaign that will take place at the JBL Ranch on September 27. "Our family started something called Happy Heart Week, that is an awareness and fundraising campaign all around Barth Syndrome. 13 years now, and all the funds go directly to the foundation to support everything that they're doing," she said.
The Sun
30-05-2025
- Business
- The Sun
Stealth Bio to cut jobs after FDA rejects therapy for rare disorder
PRIVATELY held Stealth BioTherapeutics said on Thursday the U.S. Food and Drug Administration had declined to approve its therapy for an ultra-rare condition called Barth Syndrome, prompting it to cut 30% of its workforce. Barth syndrome, which is estimated to affect around 150 people in the United States, has no approved treatments. It typically affects boys and causes heart and muscle weakness, as well as delayed growth. The FDA has asked the company to resubmit its application, which Stealth said would require more capital and prompted the job cuts. The FDA did not respond to a Reuters request for comment The therapy, elamipretide, has faced several setbacks, including the FDA's refusal to accept its marketing application in 2021. The company subsequently went private in 2022. The latest setback comes after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline. The company said the rejection came 'out of the blue'. 'We don't understand why it wasn't an approval, because there isn't really a request that we resubmit new data,' said CEO Reenie McCarthy. 'I think our frustration is: why didn't this come sooner?' McCarthy said. In October, a panel of advisers to the FDA voted 10-6 in favor of the therapy. Stealth had submitted data from a mid-stage trial that showed improvement in knee muscle strength by over 45%, which correlated with improvements on the six-minute walk test. FDA raised the possibility of an accelerated approval based on the findings, but is hesitant to extend the pathway to critically ill newborns, who make up nearly two-thirds of the therapy's expanded access program participants. The company said it will meet with the FDA next month.
Globe and Mail
14-04-2025
- Business
- Globe and Mail
Mitochondrial Myopathies Treatment Market 2032: EMA, PDMA, FDA Approval, Clinical Trials, Epidemiology, Revenue, Statistics, Therapies, and Companies by DelveInsight
Mitochondrial Myopathies Companies are Stealth BioTherapeutics, Reneo Pharmaceuticals, Abliva AB, Omeicos Therapeutics, Khondrion BV, Tisento Therapeutics, UCB BIOSCIENCES, Inc., and others. (Albany, USA) DelveInsight's ' Mitochondrial Myopathies Market Insights, Epidemiology, and Market Forecast-2032 ″ report offers an in-depth understanding of the Mitochondrial Myopathies, historical and forecasted epidemiology as well as the Mitochondrial Myopathies market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan. The Mitochondrial Myopathies market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Mitochondrial Myopathies market size from 2019 to 2032, segmented by seven major markets. The Report also covers current Mitochondrial Myopathies treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Mitochondrial Myopathies market. To Know in detail about the Mitochondrial Myopathies market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Mitochondrial Myopathies Market Forecast Some of the key facts of the Mitochondrial Myopathies Market Report: · The Mitochondrial Myopathies market size is anticipated to grow with a significant CAGR during the study period (2019-2032) · In December 2023, Reneo Pharmaceuticals, based in the US, intends to implement cost-cutting measures, including reducing its workforce by 70%, following the Phase III trial failure of its leading candidate mavodelpar. The Phase III STRIDE trial (NCT04535609) assessed mavodelpar in adult patients with primary mitochondrial myopathies (PMM). Unfortunately, the drug did not show a significant improvement in the distance walked during the 12-minute walk test (12MWT) at week 24, which was the primary endpoint of the study. · In Septemer 2023, Stealth BioTherapeutics Inc., a biotechnology company advancing treatments for diseases linked to mitochondrial dysfunction, has announced the completion of target enrollment in the NuPOWER (SPIMD-301) study for patients with primary mitochondrial myopathy (PMM). The study will continue enrolling participants through the end of the month to accommodate additional interested patients. · According to Bottoni et al. (2023), the estimated prevalence of mitochondrial diseases in children ranges from 4.7 to 15 per 100,000. · According to research conducted by Ibayashi et al. (2023) in Japan, there were 3,629 patients diagnosed with mitochondrial diseases, with a prevalence of 2.9 per 100,000 in the general population. The distribution of females to males was 53 to 47, and the most common age group affected was between 40 and 49 years old. · According to the American Academy of Ophthalmology (2023), up to 60% of cases of mitochondrial chronic progressive external ophthalmoplegia (CPEO) are attributed to deletions in mitochondrial DNA (mtDNA). Other cases result from defects in nuclear DNA (nDNA) affecting mtDNA maintenance, such as mutations in genes like POLG1, ANT, C10orf2/twinkle, or POLG2. Sporadic cases of CPEO are linked to new mutations in mtDNA, while autosomal dominant or recessive inheritance patterns suggest mutations in nuclear DNA. · Key Mitochondrial Myopathies Companies: Stealth BioTherapeutics, Reneo Pharmaceuticals, Abliva AB, Omeicos Therapeutics, Khondrion BV, Tisento Therapeutics, UCB BIOSCIENCES, Inc., and others · Key Mitochondrial Myopathies Therapies: Elamipretide, REN001, KL1333, OMT-28, Sonlicromanol, zagociguat, doxecitine and doxribtimine, and others · The Mitochondrial Myopathies market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Mitochondrial Myopathies pipeline products will significantly revolutionize the Mitochondrial Myopathies market dynamics. Mitochondrial Myopathies Overview Mitochondrial Myopathies are a group of neuromuscular diseases caused by defects in mitochondria—the energy-producing components of cells. Mitochondrial Myopathies symptoms can vary widely but often include muscle weakness, exercise intolerance, neurological issues, and fatigue. Mitochondrial Myopathies causes stem from genetic mutations, either inherited from the mother or arising spontaneously. Mitochondrial Myopathies diagnosis involves clinical evaluation, muscle biopsy, genetic testing, and biochemical analysis. Mitochondrial Myopathies treatment is largely supportive, focusing on managing symptoms and slowing progression. While there is no definitive cure, Mitochondrial Myopathies management may include physical therapy, vitamin and coenzyme supplementation, and specialized dietary approaches. Mitochondrial Myopathies in children may present differently than in adults, often with more severe manifestations. Mitochondrial Myopathies research is ongoing to discover targeted therapies and improve quality of life. Mitochondrial Myopathies prognosis varies depending on the specific mutation and severity of symptoms. Mitochondrial Myopathies awareness is growing among healthcare providers, leading to earlier detection and intervention. Mitochondrial Myopathies support groups and advocacy organizations play a key role in patient education and research funding. Mitochondrial Myopathies specialists such as neurologists and geneticists are crucial for accurate diagnosis and care planning. Mitochondrial Myopathies require a multidisciplinary approach to optimize patient outcomes. Mitochondrial Myopathies Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Mitochondrial Myopathies Epidemiology Segmentation: The Mitochondrial Myopathies market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: · Total Prevalence of Mitochondrial Myopathies · Prevalent Cases of Mitochondrial Myopathies by severity · Gender-specific Prevalence of Mitochondrial Myopathies · Diagnosed Cases of Episodic and Chronic Mitochondrial Myopathies Download the report to understand which factors are driving Mitochondrial Myopathies epidemiology trends @ Mitochondrial Myopathies Epidemiology Forecast Mitochondrial Myopathies Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Mitochondrial Myopathies market or expected to get launched during the study period. The analysis covers Mitochondrial Myopathies market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Mitochondrial Myopathies Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. The market for mitochondrial myopathies is influenced by several drivers and barriers. On the driving side, increased awareness and understanding of mitochondrial disorders have led to earlier diagnoses, expanding the patient pool seeking treatments. Advancements in genetic research and biotechnology have spurred the development of novel therapeutic approaches, attracting investment from pharmaceutical companies. Additionally, government initiatives and funding for rare diseases have provided financial support for research and development in this area. However, the market faces significant barriers, including the complexity of mitochondrial diseases, which complicates the development of effective treatments. The high costs associated with research and clinical trials for rare diseases can deter investment, and the small patient population limits potential revenue, making it less attractive for some companies. Furthermore, regulatory challenges specific to rare diseases can delay the approval and commercialization of new therapies. Balancing these drivers and barriers is crucial for stakeholders aiming to advance the mitochondrial myopathies market. Mitochondrial Myopathies Therapies and Key Companies · Elamipretide: Stealth BioTherapeutics · REN001: Reneo Pharmaceuticals · KL1333: Abliva AB · OMT-28: Omeicos Therapeutics · KL1333: Abliva AB · Sonlicromanol: Khondrion BV · zagociguat: Tisento Therapeutics · doxecitine and doxribtimine: UCB BIOSCIENCES, Inc. Mitochondrial Myopathies Market Strengths · Growing recognition of mitochondrial myopathies among healthcare professionals and the general public is leading to improved diagnosis rates and access to care · Ongoing research into mitochondrial function and disease mechanisms is leading to potential breakthroughs in treatment options and management strategies Mitochondrial Myopathies Market Opportunities Continued research into mitochondrial function and disease mechanisms may lead to the development of targeted therapies aimed at restoring mitochondrial function or mitigating disease progression Scope of the Mitochondrial Myopathies Market Report · Study Period: 2019–2032 · Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan] · Key Mitochondrial Myopathies Companies: Stealth BioTherapeutics (NASDAQ: MITO), Reneo Pharmaceuticals (NASDAQ: RPHM), Abliva AB (STO: ABLI), Omeicos Therapeutics (Private), Khondrion BV (Private), Tisento Therapeutics (Private), UCB BIOSCIENCES, Inc. (EBR: UCB), and others. · Key Mitochondrial Myopathies Therapies: Elamipretide, REN001, KL1333, OMT-28, Sonlicromanol, zagociguat, doxecitine and doxribtimine, and others · Mitochondrial Myopathies Therapeutic Assessment: Mitochondrial Myopathies current marketed and Mitochondrial Myopathies emerging therapies · Mitochondrial Myopathies Market Dynamics: Mitochondrial Myopathies market drivers and Mitochondrial Myopathies market barriers · Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies · Mitochondrial Myopathies Unmet Needs, KOL's views, Analyst's views, Mitochondrial Myopathies Market Access and Reimbursement To know more about Mitochondrial Myopathies companies working in the treatment market, visit @ Mitochondrial Myopathies Clinical Trials and Therapeutic Assessment Table of Contents 1. Mitochondrial Myopathies Market Report Introduction 2. Executive Summary for Mitochondrial Myopathies 3. SWOT analysis of Mitochondrial Myopathies 4. Mitochondrial Myopathies Patient Share (%) Overview at a Glance 5. Mitochondrial Myopathies Market Overview at a Glance 6. Mitochondrial Myopathies Disease Background and Overview 7. Mitochondrial Myopathies Epidemiology and Patient Population 8. Country-Specific Patient Population of Mitochondrial Myopathies 9. Mitochondrial Myopathies Current Treatment and Medical Practices 10. Mitochondrial Myopathies Unmet Needs 11. Mitochondrial Myopathies Emerging Therapies 12. Mitochondrial Myopathies Market Outlook 13. Country-Wise Mitochondrial Myopathies Market Analysis (2019–2032) 14. Mitochondrial Myopathies Market Access and Reimbursement of Therapies 15. Mitochondrial Myopathies Market Drivers 16. Mitochondrial Myopathies Market Barriers 17. Mitochondrial Myopathies Appendix 18. Mitochondrial Myopathies Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Ankit Nigam Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:
